Science: Novel gene therapies targeting overactive brain cells hold promise for treating neurological disorders
Time of Update: 2023-01-05
To develop the new gene therapy, the authors screened several genes known to be activated in response to stimuli and coupled their promoters to potassium channels selected for reducing nerve cell firing.
com)Resources:Yichen Qiu et al.
Express creates multiple firsts
Time of Update: 2022-08-15
Today, Caribou Biosciences announced that its CRISPR-based gene editing-engineered ready-to-use CAR-T therapy CB-010 has achieved positive results in a Phase 1 clinical trial in patients with relapsed/refractory B-cell non-Hodgkin lymphoma .
Nature Biotechnology unveils a more efficient RNA editing technique
Time of Update: 2022-03-07
To overcome these problems, Mali and colleagues designed a new guide RNA that recruits the cell's own ADARs very efficiently to edit precise target RNA regions .
【AACR Sub-Journal】An important bacterial protein related to colorectal cancer has been discovered, which is expected to develop preventive drugs
Time of Update: 2022-02-23
According to researchers at the Bloomberg School of Public Health, bacterial strains that cause common symptoms of food poisoning often contain a toxin that damages DNA in intestinal cells, potentially triggering colon cancer .
Circulation: The Changxing team of West Lake University uses base editing to bring hope to cure Duchenne muscular dystrophy
Time of Update: 2021-12-04
Recently, Life Sciences, University of the West Lake Chang Xing research group in Circulation published online entitled: Therapeutic Exon Skipping Via A CRISPR-Guided Cytidine Deaminase rescues dystrophic an In Vivo Cardiomyopathy research papers .
"Science Advances" Live Skin DNA Transfection Platform: Similar to "Cupping" of Traditional Chinese Medicine
Time of Update: 2021-11-11
This method provides an easy-to-use, cost-effective and highly scalable platform for the laboratory transfection requirements and clinical applications of nucleic acid therapy and vaccines .
Nature Sub-Journal: Transforming bacteria into a drug delivery system is expected to overcome solid solid tumors
Time of Update: 2021-11-04
The research developed a non-toxic, bacteria-based delivery system-Salmonella, which not only can easily enter cells, but also can specifically target cancer cells to directly deliver proteins (drugs) without affecting healthy cells .
Received US$3 billion from Roche to help develop gene therapies for diseases such as Alzheimer's disease
Time of Update: 2021-09-13
Today, Shape Therapeutics announced that it has reached a research and development cooperation and licensing agreement with Roche .
It will use its RNA editing technology platform RNAfix and AAVid technology platform to develop gene therapies for the treatment of Alzheimer's disease, Parkinson's disease and rare diseases .
Hepatology makes new progress
Time of Update: 2021-08-07
The study found that sorting connexin 8 (SNX8) is a new inhibitor of fatty acid synthesis through systematic screening, revealing the important function and mechanism of SNX8 in regulating non-alcoholic fatty liver disease (NAFLD) and providing a new target for NAFLD treatment And potential new strategies (click to read).
Neurology-Strengthening blood pressure leads to kidney damage and worse prognosis
Time of Update: 2021-08-03
In patients with ICH, the second acute intracerebral hemorrhage intensive blood pressure test (INTERACT2) showed that compared with the standard, intensive blood pressure had a moderate improvement in functional results; its post-mortem analysis showed that the effects of early intensive blood pressure reduction were different It is consistent in eGFR .
Significantly reduce the annual bleeding rate, two hemophilia A gene therapies show long-term effects
Time of Update: 2021-07-28
The latest published results showed that 7 patients with severe hemophilia A who received gene therapy at a dose of 6e13 vg/kg had an average annual bleeding rate (ABR) of 6 patients from baseline at a median follow-up time of 5.
Genetron Health and Siemens Healthcare reached a strategic cooperation to jointly promote precision diagnosis and treatment of lung cancer
Time of Update: 2021-07-06
More patients with non-small cell lung cancer (NSCLC) will bring efficient and accurate individualized diagnosis and treatment services, and promote the standardization of molecular testing in hospitals .
NEJM: The effect of anti-B cell maturation antigen CAR-T cell therapy bb2121 in the treatment of multiple myeloma
Time of Update: 2021-05-20
gov/31042825/" target="_blank" rel="noopener">Anti-BCMA CAR T-Cell Therapy bb2121 in Relapsed or Refractory Multiple Myeloma .
gov/31042825/" target="_blank" rel="noopener">Anti-BCMA CAR T-Cell Therapy bb2121 in Relapsed or Refractory Multiple Myeloma in this message
Roche Huntington’s ASO Phase III clinical trial stopped
Time of Update: 2021-04-27
Tominersen is an anti-nucleic acid drug that binds to Huntington mRNA and degrades this mRNA through the RNA degradation system, thus reducing the expression of toxic proteins, which may change the course of the disease.
FDA approves Vertex clinical test stem cell-derived fully differentiated islet cell therapy
Time of Update: 2021-03-12
Vertex plans to launch a phase 1/2 single-arm, open-label clinical trial in the first half of 2021, using VX-880 to treat patients with type 1 diabetes (T1D) with impaired and severe hypoglycemia.